Ikaria Drug Fails in Big Trial of Babies With Lung Disease

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Ikaria has some disappointing news out today on its lead drug development candidate. The Clinton, NJ-based biotech company, which has research and development operations in Seattle, said over the weekend that its inhaled form of nitric oxide didn’t work for premature infants with bronchopulmonary disease.

Results from a Phase III study of 800 premature infants found that Ikaria’s drug, INOT27, didn’t perform any better than standard treatments in a control group, researchers said at the European Academy of Pediatrics meeting in Nice, France.

“These results were quite unexpected based on prior evidence indicating the efficacy of inhaled nitric oxide in the prevention of bronchopulmonary disease in pre-term infants,” said Jean-Christophe Mercier, a professor of pediatrics at the University of Paris, and the study’s lead researcher, in an Ikaria statement. Mercier noted that the study called for using a low dose of nitric oxide for infants born at a higher weight, which may have contributed to the failure of the study.

Ikaria currently markets inhalable nitric oxide as INOmax, for newborns with “Blue Baby” syndrome, known medically as persistent pulmonary hypertension in newborns, a disease in which blood flow is restricted to the lungs, harming a baby’s ability to breathe. The company has attracted even greater attention for a program in earlier stages of development, a liquid sodium sulfide for injection, that is designed to induce “hibernation-on-demand.” This sort of drug is thought to hold huge promise, with one idea being that inducing a metabolic slowdown could buy time for a surgeon trying to save someone before he or she bleeds to death.

Ikaria’s Csaba Szabo told me last month that the company is planning to move forward with a Phase II clinical trial program of the sodium sulfide candidate, IK-1001, by the end of this year. Ikaria is a private company, so it doesn’t report its finances publicly, but we’ll find out if it’s still planning to go full-steam ahead with the IK-1001 program, or if it needs to conserve cash in the wake of the Phase III failure with its lead drug candidate.